专利检索 ap:("Isis Pharmaceuticals, Inc." OR "University of Rochester") AND inv:"Freier Susan, M" 第 1 页

1.

发明公开
MODULATION OF DYSTROPHIA MYOTONICA-PROTEIN KINASE (DMPK) EXPRESSION 审中-公开
标题翻译：强直性肌营养不良蛋白激酶的调制（DMPK）表达

公开(公告)号：EP2595663A2

公开(公告)日：2013-05-29

申请号：EP11810291.2

申请日：2011-07-19

申请人： Isis Pharmaceuticals, Inc. , University of Rochester

发明人： Bennett C. Frank , Freier Susan, M , Macleod, Robert, A , Pandey, Sanjey, K , Thornton, Charles, A , Wheeler, Thurman , Cheng, Seng, H. , Leger, Andrew , Wentworth, Bruce, M.

IPC分类号： A61K48/00

CPC分类号： C12N15/1137 , C12N15/113 , C12N2310/11 , C12N2310/315 , C12N2310/3181 , C12N2310/321 , C12N2310/3231 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , C12N2310/3525 , C12Y207/11 , C12N2310/322 , C12N2310/3533 , C12N2310/3527

摘要： Provided herein are methods, compounds, and compositions for reducing expression of a DMPK mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for preferentially reducing CUGexp DMPK RNA, reducing myotonia or reducing spliceopathy in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate type 1 myotonic dystrophy, or a symptom thereof.